Use of Sotatercept in France since Its Availability in June 2024: A Nationwide Study from the French Health Data System (SNDS)

Pulmonary arterial hypertension (PAH) is a rare, severe and progressive disease associated with poor quality of life and high mortality. Sotatercept, a first-in-class fusion protein, has recently been shown to provide substantial clinical benefit as add-on therapy in PAH and was made available in France in 2024 through an early access program, with eligibility criteria that progressively broadened over time. We aimed to describe the characteristics of the first patients treated with sotatercept in France, its real-world administration patterns, and associated changes in background PAH therapy. Using the French National Early Access and Expensive Drugs Cohort (AP-MO), derived from the French Health Data System (SNDS), we included all patients who initiated sotatercept since the start of the early access program and followed them until 31 October 2025. Baseline characteristics included sex, age, social deprivation index, comorbidities, and time since treatment initiation. Changes in background PAH therapy were also described. We identified 257 patients treated with sotatercept (median age 53 years; 75.5% women; median follow-up 8.9 months). Among patients with ≥ 90 days follow-up ( n = 205), background therapy remained unchanged in 67% of patients. Most administrations followed the expected 21-day interval, and 94.2% received at least one dose renewal. Thirteen deaths occurred during follow-up (0.55 per 100 person-months), and two patients underwent lung transplantation. These findings provide the first real-world, nationwide insights into sotatercept use in routine clinical practice, showing treatment patterns broadly consistent with initial recommendations, including maintenance of background therapy and adherence to the recommended dosing interval. Further studies are warranted to assess the long-term effectiveness and safety of sotatercept.